Camp4 Therapeutics Corporation · NASDAQ:CAMP
Camp4 Therapeutics — Investor Presentation (January 2025)
32 slides from Camp4 Therapeutics's investor presentation, Jan 7, 2025
Slide 1: Corporate Overview January 2025
Pioneering a new class of RNA medicines to increase targeted gene expression Corporate Overview January 2025
Slide 2: Forward-Looking Statements
Forward - Looking Statements This presentation contains forward - looking statements that are based on the beliefs and assumptions and on information currently available to CAMP4’s management. All statements other than statements of historical fact contained in this presentation are forward - lookin
Slide 3: CAMP4 is the leader in regRNA discovery and regRNA-targeting ASO therapies
CAMP4 is the leader in gene regulatory RNA ( regRNA ) discovery and regRNA - targeting antisense oligonucleotide (ASO) therapies to upregulate gene expression to restore healthy protein levels Pioneering a new class of RNA medicines to increase targeted gene expression 3 We built our Proprietary RAP
Slide 4: World-class management team, experienced board and advisors
World - class management team, experienced board and advisors Josh Mandel - Brehm President & CEO David Bumcrot , PhD Chief Scientific Officer Michelle Gates Chief People Officer Caleb Moore Chief Business Operations Officer Satya Kuchimanchi , PhD SVP, Technical Operations Alla Sigova, PhD VP, Head
Slide 5: Advancing a pipeline in metabolic and CNS genetic diseases
Commercial Rights Anticipated Milestones Phase 3 Phase 2 Phase 1 Discovery & Preclinical Target Indication Program Metabolic diseases Phase 1 MAD data in 2H’25 CPS1 Urea Cycle Disorders CMP - CPS - 001 CNS diseases Initiation of GLP tox studies in ‘25 SYNGAP1 SYNGAP1 - related Disorders CMP - SYNGAP
Slide 6: Agenda
6 Agenda CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1 - related Disorders
Slide 7: regRNAs play a central role in the regulation of every gene's expression
regRNAs play a central role in the regulation of every gene’s expression 7 mRNA Activators and repressors bind to regRNAs to control the transcriptional state Source: Sharp, Chakraborty, Henninger & Young, RNA 2022; Henninger, Oksuz , Shrinivas et al., Cell 2021; Sartorelli & Lauberth , NatStructMol
Slide 8: CAMP4's proprietary RAP Platform catalogs thousands of regRNAs and generates ASO leads
8 1. Map candidate regRNAs 2. Generate ASO leads 3. Optimize leads CAMP4’s proprietary RAP Platform catalogs thousands of regRNAs in any tissue and generates ASO leads to increase gene expression Next - gen sequencing and machine learning algorithms map regRNAs controlling every expressed gene. One
Slide 9: CAMP4 applies its RAP Platform to genetic diseases where modest increases in expression matter
CAMP4 applies its RAP Platform to genetic diseases where modest increases in gene expression can be clinically meaningful 9 Clinical and Regulatory • Defined patient population • Efficacy can be evaluated in Ph 1/2 based on availability of biomarkers • Clear or established path to approval Platform
Slide 10: RAP Platform has potential to address a broad array of prevalent diseases
10 RAP Platform has potential to address a broad array of prevalent diseases Gene upregulation Parkinson’s disease FTD, ALS, LB dementia Alzheimer's disease Fibrosis MASH Friedreich ataxia FTD, ALS DMD SMA Sickle cell disease Fibrosis Haploinsufficient Paralogs Repeat Expansion Metabolic Enzyme Repl
Slide 11: Agenda
11 Agenda CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1 - related Disorders
Slide 12: Urea Cycle Disorders are life-threatening inherited metabolic diseases
Urea Cycle Disorders (UCDs) are a set of life - threatening inherited metabolic diseases characterized by the accumulation of toxic ammonia 12 OTC ASS1 ASL ARG1 CPS1 Dysfunctional Urea Cycle NAGS^ OTC mutated retaining only partial activity UREA AMMONIA Mutation in one of several urea cycle enzymes
Slide 13: CMP-CPS-001 targets increased expression of CPS1
CMP - CPS - 001 i s a GalNAc - conjugated ASO that binds to a CPS1 - specific regRNA to increase CPS1 expression and upregulate the expression of multiple urea cycle enzymes to amplify the conversion of ammonia to urea, potentially addressing more than 90% of patients with late onset UCDs. CAMP4 is
Slide 14: Modest increases in ureagenesis resulted in significant reductions in disease severity
14 Clinical observations and precedent have found that modest increases in ureagenesis resulted in significant reductions in disease severity Early onset (Mortality at birth) Severe UCDs (~90% patients) OTC female heterozygotes (Range of symptoms) WT Late onset patients (>1 month); >5,000 U.S. patie
Slide 15: CMP-CPS-001 has the potential to be the first disease modifying pan-UCD therapy
15 CMP - CPS - 001 has the potential to be the 1 st disease modifying pan - UCD therapy Increase ureagenesis via GalNAc - conjugated ASO *CMP - CPS - 001 has been granted a Rare Pediatric Designation and Orphan Drug Designation 1x monthly (titratable), subcutaneous • ~90% of severe UCD patients (ped
Slide 16: CMP-CPS-001 potential first disease-modifying therapy by increasing ureagenesis
• Biomarker: Urea cycle activity (ureagenesis) can be monitored in healthy volunteers and patients using the ureagenesis rate test (URT) • Phase 1 study: CAMP4 is utilizing URT in SAD / MAD Phase 1 CMP - CPS - 001 clinical study • Proof of concept: Multiple companies have utilized the URT in healthy
Slide 17: CMP-CPS-001 targets the key regRNA controlling CPS1 expression
0 2 4 6 UTC 1.25 μ M 2.5 μ M 5 μ M 0.156 μ M 0.313 μ M 0.625 μ M 1.25 μ M 2.5 μ M 5 μ M Fold change of CPS1 mRNA levels (relative to UTC) 17 CMP - CPS - 001 targets the key regRNA controlling CPS1 expression RAP Platform identified CPS1 regRNA CMP - CPS - 001 increases CPS1 mRNA in primary WT liver
Slide 18: ASO targeting mouse Cps1 regRNA in Otc-deficient mice reduces ammonia
ASO targeting mouse Cps1 regRNA in Otc - deficient mice reduces ammonia and supports once - monthly dosing 18 Onset Maintenance Return (1 - 2 wks ) (~4 wks ) (~1 - 2 wks ) • The Otc spf - ash mouse model carries a patient mutation in Otc that reduces mRNA levels • Otc activity is 5% - 10% of wild -
Slide 19: CMP-CPS-001 increases ureagenesis in WT mice with humanized liver
CMP - CPS - 001 increases ureagenesis in WT mice with humanized liver Data confirm target engagement of CMP - CPS - 001 in human hepatocytes in vivo and show increased expression of other urea cycle enzymes 19 0.0 0.5 1.0 1.5 2.0 NAGS CPS1 OTC ASS1 ASL ARG1 Relative expression (normalized to PBS) 14
Slide 20: CMP-CPS-001 increases ureagenesis in NHPs
20 Using 13 C - Sodium Acetate for ureagenesis in WT NHP NHP administered drug once - monthly. Ureagenesis assessed up to 30 days after final dose. Data shown is one week after a second d ose. Error bars represent standard error of the mean; PBS denotes phosphate - buffered saline; * denotes p<.05;
Slide 21: Ongoing CMP-CPS-001 Phase 1 SAD/MAD clinical trial in healthy volunteers
21 Ongoing CMP - CPS - 101 Phase 1 SAD / MAD clinical trial in healthy volunteers n = 10 - 12 participants per cohort Cohort 1 Cohort 1 (.2 mg / kg) Cohort 2 (.6 mg / kg) Cohort 3 (1.8 mg / kg) Cohort 4 (4 mg / kg) Cohort 2 q monthly x 3 Cohort 3 Cohort 4 SAD portion of Phase 1 MAD portion of Phase
Slide 22: SAD portion of study completed; safety results
• SAD portion of study completed. Conducted planned interim analysis of safety data for 48 normal healthy volunteer participants. • 4 Cohorts of 12 participants each, randomized 3:1 investigational product to placebo; 32 individuals received CMP - CPS - 001. • CMP - CPS - 001 has been well tolerated
Slide 23: Phase 1 has the potential to enable a combined Phase 2/3 registration study in 2026
23 Phase 1 has the potential to enable a combined Phase 2/3 registration study anticipated in 2026 Safety Pharmacokinetics Ureagenesis rate test (PD) • Vitals • Cardiac monitoring • Liver function tests • Immunogenicity • Plasma and urine measurements • Compare human pharmacokinetic behavior to pre
Slide 24: Agenda
24 Agenda CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1 - related Disorders
Slide 25: SYNGAP1-related disorders, a severe genetic neurodevelopmental condition
SYNGAP1 - related disorders, a severe genetic neurodevelopmental condition 25 Mutations in SYNGAP1 lead to decreased SYNGAP protein, causing increased synaptic firing • Highly burdensome symptom array: • Intellectual disability, severe behavioral problems, ASD • Generalized epilepsy • Sleep problems
Slide 26: Restoring SYNGAP1 levels to treat disease
26 SYNGAP1 AMPAR at synapse Neuronal Excitability CAMP4 aims to increase SYNGAP1 protein levels to restore SYNGAP1 at the synapse and improve disease symptoms The CMP - SYNGAP program has identified lead ASOs that bind to a SYNGAP1 - specific regRNA to increase SYNGAP1 expression. Intrathecal admini
Slide 27: CAMP4 positioned as the only transcriptional-level ASO approach
CAMP4 positioned as the only transcriptional - level ASO approach 27 Discovery Preclinical Clinical Trials AAV Delivered tRNA Read - through premature stop codon, limited data ASO targeting pre - mRNA (splicing) to prevent NMD AAV Delivered dCas Target regulatory elements, limited info No industry s
Slide 28: Targeting SYNGAP1 regRNA increased expression of mRNA and protein in vitro
Targeting SYNGAP1 regRNA demonstrated 2 - fold increased expression of SYNGAP1 mRNA and protein in vitro 28 ASO binding to SYNGAP1 regRNA d irectly i ncreases transcription ASOs targeting regRNA restore wildtype SYNGAP1 mRNA levels in iPSC neurons A B C Mapped SYNGAP1 regRNA in iPSC neurons 1 2 3 50
Slide 29: ASOs targeting SYNGAP1 regRNA increased mRNA and protein in vivo
ASOs targeting SYNGAP1 regRNA demonstrated dose - responsive increase in mRNA and protein in vivo 29 Dose - responsive increase in SYNGAP1 protein with l ead ASO in humanized mouse model Dose - responsive increase in Syngap1 mRNA with mouse surrogate ASO 2024-063 P21 ICV 2-week post dose CO-19429 Do
Slide 30: New discovery program: Increasing GBA1 expression for Parkinson's disease
• GCase and - synuclein (hallmark disease protein for PD) are part of a positive feedback loop 4 • GCase activity also reduced in sporadic PD 3 • Plan to explore therapeutic potential in both genetically defined GBA - PD and sporadic PD 30 New discovery program: Increasing GBA1 expression for the po
Slide 31: CAMP4 is the leader in regRNA discovery and regRNA-targeting ASO therapies
CAMP4 is the leader in gene regulatory RNA ( regRNA ) discovery and regRNA - targeting antisense oligonucleotide (ASO) therapies to upregulate gene expression to restore healthy protein levels Pioneering a new class of RNA medicines to increase targeted gene expression 31 We built our Proprietary RA
Slide 32: Thank you
Thank you
CAMP4 Therapeutics — Investor Presentation (January 2025) on 6ix